A combined AI and cell biology approach surfaces targets and mechanistically distinct Inflammasome inhibitors

Summary: Inflammasomes are protein complexes that mediate innate immune responses whose dysregulation has been linked to a spectrum of acute and chronic human conditions, which dictates therapeutic development that is aligned with disease variability. We designed a scalable, physiologic high-content...

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Main Authors: Daniel Chen, Tempest Plott, Michael Wiest, Will Van Trump, Ben Komalo, Dat Nguyen, Charlie Marsh, Jarred Heinrich, Colin J. Fuller, Lauren Nicolaisen, Elisa Cambronero, An Nguyen, Christian Elabd, Francesco Rubbo, Rachel DeVay Jacobson
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:iScience
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Online Access:http://www.sciencedirect.com/science/article/pii/S2589004224026294
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Summary:Summary: Inflammasomes are protein complexes that mediate innate immune responses whose dysregulation has been linked to a spectrum of acute and chronic human conditions, which dictates therapeutic development that is aligned with disease variability. We designed a scalable, physiologic high-content imaging assay in human PBMCs that we analyzed using a combination of machine-learning and cell biology methods. This resulted in a set of biologically interpretable readouts that can resolve a spectrum of cellular states associated with inflammasome activation and inhibition. These methods were applied to a phenotypic screen that surfaced mechanistically distinct inflammasome inhibitors from an annotated 12,000 compound library. A set of over 100 inhibitors, including an array of Raf-pathway inhibitors, were validated in downstream functional assays. This approach demonstrates how complementary machine learning-based methods can be used to generate profiles of cellular states associated with different stages of complex biological pathways and yield compound and target discovery.
ISSN:2589-0042